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Grant to fund gene therapy study at Allegheny General Hospital

| Thursday, Aug. 9, 2012, 12:34 p.m.

Researchers at Allegheny General Hospital hope a $1.7 million federal grant will help develop new gene therapies to treat a salivary gland problem that affects millions of people.

A team led by Michael Passineau, director of the Gene Therapy Program at the hospital's Allegheny Singer Research Institute, will use the four-year grant from the National Institutes of Health to transfer therapeutic DNA into pig cells using ultrasound. Usually, the transfer is conducted using a virus.

“What makes it interesting is that we're grabbing off-the-shelf technologies, using elements that are already approved by the Food and Drug Administration,” Passineau said on Thursday. “We've taken a bunch of things out there and combined them, and that gives us a new effect.”

Gene therapy involves the use of DNA to treat disease, introducing new genes to a patient's cells to replace missing or malfunctioning genes. Although viruses are used to introduce the DNA into cells, a person's immune system can hamper that process.

Passineau said dozens of patients within the West Penn Allegheny Health System, AGH's parent, have xerostomia, a complication from radiation used to treat head and neck cancer in patients with damaged salivary glands. That damage leads to speech, chewing and swallowing problems.

Also, Passineau said, more than 4 million people suffer from Sjogren's syndrome, an autoimmune disease that damages moisture-producing glands with white blood cells.

Passineau said researchers, including AGH radiation oncologist Dr. Mark Trombetta and cardiologist Dr. Robert Biederman, will use pigs in their tests. He said researchers already have used rodents. If successful in pigs, Passineau added, the technique could go straight to human testing.

Aside from stopping Sjogren's syndrome, Passineau said, researchers could develop a “super saliva” to help elderly patients who can't brush their teeth regularly by developing a gene that would prevent bacteria from sticking to a person's teeth.

“I think it represents the next evolution of gene transfer technology,” Passineau said. He added that the technique, if successful, could be used to treat other organs, including the heart, pancreas and kidneys.

Bill Vidonic is a staff writer for Trib Total Media. He can be reached at 412-380-5621 or bvidonic@tribweb.com.

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